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Epidermolysis Bullosa - Healing Genes
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Epidermolysis Bullosa

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Topical QR-313 in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Due to Mutation(s) in Exon 73 of the COL7A1gene (WINGS)

A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene


Phase 1/2
DESCRIPTION:

Four medical centers in CA, CO, MN, and OH are recruiting participants who have specific types of Recessive Dystrophic Epidermolysis Bullosa to test a new, topical treatment. The treatment aims to slow progression of EB wounds through a drug that binds to and stops the cellular messaging system that causes inflammation.

The actual drug and a placebo will be applied 2 to 3x per week to two wound areas, as assigned by the study, for 4 weeks. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.


PATIENT MUST:

  • Be 6 years of age or older
  • Have at least 1 target wound area
  • Have life expectancy > 6 months

THE STUDY INVOLVES:

  1. Screening before the treatment.
  2. Visit to the study center for assignment of the drug and/or placebo to the targeted wounds.
  3. For 4 weeks, the medicines will be applied to the assigned wounds 2 to 3 times per week.
  4. Home and site visits will follow for regular assessments up to 8 weeks post the last dose.

LOCATIONS AND CONTACTS:

The study locations include:

Stanford University School of Medicine, LPCH in Palo Alto, CA

Contact: Kunju Sridhar  |  650-721-4902  |  [email protected]

Children’s Hospital Colorado in Aurora, CO

Contact: Kathleen Peoples  |  720-777-4708 |   [email protected]

Journey Clinic, Center for Pediatric Blood and Marrow Transplantation in Minneapolis, MN

Contact: Christen Ebens, MD  |  612-626-8094  |  [email protected]

Cincinnati Children’s Hospital in Cincinnati, OH

Contact: Bret Augsburger  |  513-803-9009  |  [email protected]

SPONSOR INFORMATION:

ProQR Therapeutics

Or go online:

https://clinicaltrials.gov/ct2/show/NCT03605069

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